Yesterday was a day to celebrate biotech! 🥳
Congrats to Vertex and CRISPR Therapeutics on FDA approval of CASGEVY
• 1st product that uses gene editing (See Exa-cel below for more info)
And Bluebird on FDA approval of Lyfgenia, another autologous stem cell-based gene therapy to treat Sickle cell disease, approved today too!
Interesting that both were approved on a single-arm (no control arm) trial, as the FDA is more willing to accept this data for a rare genetic disease with a high unmet need, and a strong biological basis.
From the FDA press release, it seems that Lyfgenia will have a Black-Box safety warning (re: hematologic malignancies), but not CASGEVY (safety data looked impressive)
Two more approved cell therapy products,
helping solve medical challenges
biopharma will continue to find even better therapies that are easier to tolerate/available to more sickle cell patients with similar efficacy.
And Dx companies will help to ID the small number of patients that won't respond to CASGEVY
We know our therapies and Dx are not perfect...
But over time, we are giving more people, more healthy years
Because that's what we do in biotech...
Continue to find ways to make life better
Here's more on CASGEVY
What is CASGEVY?
CASGEVY (Exa-cel) is a cell therapy that uses CRISPR/Cas9 to edit the patient’s own (autologous) hematopoietic stem cells (HSCs) outside their body (ex vivo) to produce high levels of fetal hemoglobin in red blood cells after the gene edited HSCs are delivered back to the patient.
An excellent option for many sickle cell patients
• See FIG. 9 📸 for impressive efficacy data
⇨ After years of painful VOC events, CASGEVY reduces/eliminates them in almost all patients
The FDA characterized the clinical efficacy results of exa-cel to reduce VOCs in SCD patients as "strongly positive".